Gene therapy shows promise for rare eye disease that causes blindness
NCT ID NCT03293524
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision over 1.5 years, with follow-up up to 5 years. The goal was to see if GS010 could improve or stabilize eyesight in those with vision loss for less than a year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GS010 (lenadogene nolparvovec) gene therapy
- What this could lead to
- If successful, this gene therapy could slow or partially reverse vision loss in people with a specific form of Leber hereditary optic neuropathy.
- What could go wrong
- This is a Phase 3 trial, but results may not apply to all patients. Gene therapy carries risks like injection-related complications, and long-term effects are still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHNO Les Quinze Vingts
Paris, Paris, 75012, France
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Department of Ophthalmology, Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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Doheny Eye Center UCLA Pasadena
Pasadena, California, 91105, United States
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Emory Healthcare - The Emory Clinic
Atlanta, Georgia, 30322, United States
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Hospital Universitario Ramon y Cajal
Madrid, 28034, Spain
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IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica
Bologna, 40139, Italy
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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, Greater London, EC1V 2PD, United Kingdom
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Taipei Veterans General Hospital
Taipei, 11217, Taiwan
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Universitair Ziekenhuis Gent
Ghent, 9000, Belgium
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University of Colorado Health Eye Center
Aurora, Colorado, 80045, United States
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Vanderbilt Eye Institute
Nashville, Tennessee, 37232, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Gene therapy for inherited blindness passes early safety check
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- Can a simple blood test predict blindness before it starts?
- Gene therapy shows promise in Long-Term study for rare blindness